Leukemiahttp://www.businessinsider.com/category/leukemia
en-usThu, 22 Feb 2018 00:30:09 -0500Thu, 22 Feb 2018 00:30:09 -0500The latest news on Leukemia from Business Insiderhttp://static3.businessinsider.com/assets/images/bilogo-250x36-wide-rev.pngBusiness Insiderhttp://www.businessinsider.com
http://www.businessinsider.com/military-base-drinking-water-poison-2017-4The US military is facing another polluted drinking water scandalhttp://www.businessinsider.com/military-base-drinking-water-poison-2017-4
Wed, 26 Apr 2017 18:19:00 -0400Eric Pianin
<p><img style="float:right;" src="http://static4.businessinsider.com/image/506ad3266bb3f7244000000a-946/screen shot 2012-10-02 at 74209 am.jpg" alt="camp lejeune"></p><p>For years, notorious environmental disregard at US military bases has resulted in poisoned drinking water and placed military personnel and their families at serious risk because of exposure to toxins.</p>
<p>A 2016 <a href="https://law.stanford.edu/publications/contamination-at-u-s-military-bases-profiles-and-responses/">report</a> in the Stanford Environmental Law Journal warned, "There is an epidemic of toxic contamination at US military bases." The widespread chemical contamination of soil, groundwater and nearby lakes and streams throughout the country poses threats to the health and well-being of service members, their families, and nearby civilian communities.</p>
<p>Remarkably, pollution at military bases is so widespread that more than two-thirds of the nearly 900 highly contaminated federal Superfund sites are affiliated with the military, states the report written by John W. Hamilton, a Stanford law student.</p>
<p>The problem is even more severe overseas, where the US military regularly used open-air burn pits at many operations in Iraq and Afghanistan to dispose of waste including batteries, solvents and electronics.</p>
<p>Among the worst domestic cases highlighted in the 249-page report: Camp LeJeune, N.C., the largest Marine Corps installation on the East Coast and home to about 170,000 active duty and civilian employees and retirees. The drinking water on the 240-square mile base has been poisoned for decades by "a toxic cocktail of industrial solvents, dry-cleaning chemicals and gasoline."</p>
<p>Also, the Naval Air Station Fallon in western Nevada, home to 5,000 active duty and civilian employees and a premier combat flight training center, has the dubious distinction of having been investigated as a "cancer cluster" by the Centers for Disease Control and Prevention (CDC). Sixteen children in the city were diagnosed with leukemia between 1997 and 2002, an incident rate that was far above the national average.</p>
<p>The precise cause of the leukemia outbreak has yet to be determined. However, the Fallon community has long been exposed to multiple sources of toxins, such as naturally occurring arsenic in the ground water, tungsten in the air and the water, and benzene from an oil pipeline that supplies the base’s jet fuel.</p>
<p><img src="http://static3.businessinsider.com/image/56c761ae6e97c62c008b85fd-2048/16724604222bf96d6305ek.jpg" alt="Marine corps" data-mce-source="U.S. Marine Corps photo by Lance Cpl. Christopher Mendoza/Released" data-mce-caption="U.S. Marines with Special-Purpose Marine Air-Ground Task Force Crisis Response-Africa ascend ropes during an obstacle course on Rota Naval Base, Spain, Feb. 26, 2015." data-link="https://www.flickr.com/photos/marine_corps/16724604222/"></p>
<p>Finally, the Marine Corps Air Station in El Toro, Calif., was a hub of military aviation activity until it was closed in 1999. After decades of high-tempo flight activity, the carcinogenic industrial solvent widely used on the base to clean jet engines between sorties washed onto the soil and into the groundwater supply. The base was listed as a Superfund site in 1990.</p>
<p>Now comes a controversy in northeastern Michigan where a state lawmaker is clashing with the US Air Force over responsibility for polluted drinking water near the former Wurtsmith Air Force Base near Oscoda.</p>
<p>Last year, the Michigan Department of Health and Human Services discovered perfluorinated chemical contamination in drinking water wells near the former base that threatened a nearby lake and creeks. PFCs are a group of chemicals used in fire-fighting foams, some nonstick cookware, waterproof clothing and manufacturing.</p>
<p>State officials’ concerns about pollution in the area date back to 2012, when the health department issued a "do not eat" advisory for all fish caught near Wurtsmith Air Force Base. Laboratory analysis of the fish showed levels of PFCs that were unsafe for human consumption.</p>
<p>In January, a new state law took effect saying that the military must supply safe drinking water to residents of Oscoda whose wells were polluted with toxic chemicals from Wurtsmith.</p>
<p>The law amended Michigan’s Safe Drinking Water Act to require either the federal government or the state to provide an "alternative water supply" to any property owner with a polluted well if state health officials issue a related drinking water advisory and the government was the source of the pollution.</p>
<p>Republican state Sen. Jim Stamas of Midland said he sponsored the legislation after military officials notified him last year that the Air Force would supply an alternative water supply to affected properties if the Michigan legislature passed a law requiring that, according to the <a href="https://www.usnews.com/news/best-states/michigan/articles/2017-04-24/air-force-wont-supply-clean-water-to-polluted-michigan-city">Associated Press</a>.</p>
<p><img src="http://static1.businessinsider.com/image/572227499105841c008bf8bf-1600/swimmer-military.jpg" alt="swimmer military" data-mce-source="Flickr/DVIDSHUB" data-link="https://www.flickr.com/photos/dvids/6258125311/"></p>
<p>But now the Air Force is pushing back against the new law, asserting that the military won’t provide clean water to the area because the law is "discriminatory."</p>
<p>"The Michigan law does discriminate as it only applies to federal and state agencies, not to all entities and persons," Air Force spokesman <a href="http://www.mlive.com/news/index.ssf/2017/04/usaf_michigan_pa_545.html">Mark Kinkade said</a>.</p>
<p>Kinkade says the federal Comprehensive Environmental Response, Compensation, and Liability Act (CERCLA), which created the Superfund program, only compels the US government to comply with state law if it's not discriminatory.</p>
<p>A furious Stamas replied that he was "extremely disappointed in the US Air Force" for not living up to its word and its responsibilities. He said that the federal government "needs to be held accountable" for what it did in polluting the base.</p>
<p>The Republican lawmaker said he would ask state Attorney General Bill Schuette to pursue legal action against the military to enforce the new law.</p>
<p>Schuette is no stranger to water contamination controversies. He has led the criminal and civil investigation of the lead poisoning of Flint’s drinking water supply. So far, 13 former state and local officials face criminal counts stemming from Schuette’s investigation. &nbsp;</p>
<p>The Stanford Environmental Law Journal analysis says suing the US military could be far more challenging than going after state and local officials. The federal Tort Claim Law and a slew of legal precedents make it difficult for state or local governments or veterans and their families impacted by chemical pollution from going after the military.</p><p><strong>SEE ALSO:&nbsp;<a href="http://www.businessinsider.com/this-bill-to-take-care-of-marines-exposed-to-contaminated-water-could-change-everything-for-veterans-2012-8" >The US Finally Agrees To Care For Marines Poisoned By Camp Lejune Drinking Water</a></strong></p>
<p><a href="http://www.businessinsider.com/military-base-drinking-water-poison-2017-4#comments">Join the conversation about this story &#187;</a></p> <p>NOW WATCH: <a href="http://www.businessinsider.com/grease-drain-explainer-process-sewer-fatbergs-flooding-london-2017-5">Why you should never pour grease down the drain</a></p> http://www.businessinsider.com/r-novartis-says-82-pct-of-leukemia-patients-in-remission-after-car-t-2016-12An experimental treatment eliminated an aggressive blood cancer in 82% of patients treated (NVS)http://www.businessinsider.com/r-novartis-says-82-pct-of-leukemia-patients-in-remission-after-car-t-2016-12
Mon, 05 Dec 2016 09:23:00 -0500Deena Beasley
<p><img style="float:right;" src="http://static1.businessinsider.com/image/565471b2dd089574268b45b1-800/sem_blood_cells.jpg" alt="blood cells" data-mce-source="Bruce Wetzel/Wikimedia Commons" /></p><p>(Reuters) - An experimental cancer therapy being developed by Novartis AG eliminated an aggressive form of blood cancer in 82 percent of children and young adults treated with modified immune cells in a mid-stage trial, the company said on Saturday.</p>
<p>Interim results from the multi-center trial for 50 patients with acute lymphoblastic leukemia whose cancer returned or did not respond to other treatment, showed that 41 were disease-free three months after treatment with the drug, called CTL019.</p>
<p>The trial results were presented at a meeting of the American Society of Hematology in San Diego.</p>
<p>Novartis estimated that 60 percent of those responders were relapse-free after six months. Complete remission was defined as including "remission with incomplete blood count recovery."</p>
<p>CTL019 is part of an experimental class of drugs that are made by genetically altering a patient's T-cells, a type of white blood cell, in the lab to help the immune system find and kill cancer cells. The modified cells, called chimeric antigen receptor T-cells, or CAR-T, are infused into the patient.</p>
<p>Novartis said it plans to file early next year for U.S. Food and Drug Administration approval of CTL019.</p>
<p>The company said nearly half of trial patients experienced severe cytokine release syndrome, a dangerous buildup of toxic debris known as CRS, and 15 percent experienced serious neurological problems including delirium. Researchers said the side effects were treated, and no patients died due to CRS.</p>
<p>CRS, a known side effect of CAR-T therapy, led to the decision last week to halt a trial of JCAR015, a rival drug being developed by Juno Therapeutics Inc.</p>
<p>"We have learned that the patients who come in with more leukemia in their body have a much higher risk of getting sick," said Dr. Stephan Gruff, the Novartis trial's lead investigator and director of the cancer immunotherapy program at the Children's Hospital of Philadelphia.</p>
<p>He said the trial did not show a big difference between patients whose leukemia was refractory, meaning they had stopped responding to treatment, and patients who had relapsed, or might have responded temporarily to chemotherapy. "We do see a difference in toxicity," the researcher said. "Refractory patients with high disease burden can get sicker temporarily on the way to remission."</p>
<p>(Reporting by Deena Beasley; Editing by Richard Chang)</p><p><strong>SEE ALSO:&nbsp;<a href="http://www.businessinsider.com/pfizer-and-regeneron-ceos-on-drug-pricing-and-reputation-2016-12" >Pharma CEOs got into a heated debate over why people hate the industry</a></strong></p>
<p><strong>DON'T MISS:&nbsp;<a href="http://www.businessinsider.com/digitization-radiology-2016-11" >Digital disruption in radiology — will it put doctors out of work?</a></strong></p>
<p><a href="http://www.businessinsider.com/r-novartis-says-82-pct-of-leukemia-patients-in-remission-after-car-t-2016-12#comments">Join the conversation about this story &#187;</a></p> <p>NOW WATCH: <a href="http://www.businessinsider.com/elon-musk-explains-spacex-falcon-heavy-central-core-crash-landing-2018-2">Elon Musk explains the one thing that went wrong with SpaceX's Falcon Heavy flight</a></p> http://www.businessinsider.com/colombias-battle-with-novartis-2016-5Colombia's battle with the world's biggest drugmaker could set a precedent for generic medshttp://www.businessinsider.com/colombias-battle-with-novartis-2016-5
Fri, 20 May 2016 22:22:00 -0400Alan Hernández
<p><img style="float:right;" src="http://static3.businessinsider.com/image/5628c530bd86ef135c8b8b77-1339-1004/antiretroviral arv pills medicine.jpg" alt="Antiretroviral ARV Pills Medicine" data-mce-source="REUTERS/Siphiwe Sibeko" data-mce-caption="Nine-year-old Tumelo shows off antiretroviral (ARV) pills before taking his medication at Nkosi's Haven, south of Johannesburg November 28, 2014." /></p><p>Colombia has told pharmaceutical giant Novartis that unless it lowers the price of one of its cancer medications by the end of the month, it will lose the monopoly of the market in the country that it currently enjoys.</p>
<p>The government's tough stance on the cancer medication imatinib &mdash; which is commercially sold as Gilvec or Gleevec &mdash; has been applauded by activists who say it could set a precedent that could start to improve access to expensive drugs throughout the world.</p>
<p>James Love, the director of Knowledge Ecology International, an US-based NGO that campaigns for a more equal distribution of knowledge resources, told VICE News that the Colombian effort to bring down the prices of the drug would have an impact way beyond its borders.</p>
<p>"It is going to be consequential in terms of price, especially in other South American nations, where the goal is to build a universal healthcare system," Love said. "It would mean imatinib would be more accessible than ever in other parts of the world."</p>
<p>The ultimatum culminates four years of tension over the government's plan to widen access to the medicine. In 2012 the authorities refused to give Novartis the patent to imatinib in Colombia but, later that year, a judge overturned that decision and granted the company exclusive rights over the drug in the country.</p>
<p>After the ruling, the government sought to persuade Novartis to reduce the price of the drug by 50 percent but, so far, the Swiss pharmaceutical giant has ruled this out.</p>
<p>Imatinib, which is used to treat leukemia, costs $0.10 per mg in Colombia. With dosage varying between 100mg and 800mg per day, the drug costs an average of $15,000 per year per patient &mdash; nearly double the country's per capital income. According to a study published in <em>BMJ, </em>a popular medical journal, generic imatinib could cost as little as $128 per patient per year.</p>
<p>"For us, it's a question of survival," Colombia's health minister Alejandro Gaviria told the&nbsp;<em>Associated Press</em> this week.</p>
<p>Gaviria has said that it is still possible that a deal could be reached with Novartis before the industry regulator gives the green light to forcing the end of the patent, and allowing him to issue "compulsory licenses" that would permit other pharmaceutical companies to put generic versions of imatinib on the market at a fraction of the cost. If not deal is forthcoming, this is expected to happen by the end of the month.</p>
<p class="read-more">Leaked diplomatic papers dated in late April and obtained by <em>Knowledge Ecology International </em>show that high officials in Colombia's Embassy in Washington warned the government back home that they were receiving pressure from the US Congress and the Office of the Unites States Trade Representative to pull back from its conflict with Novartis.</p>
<p><img class="center" src="http://static4.businessinsider.com/image/573dcce45124c925144baa14-800-461/novartis-launches-new-entresto-trials-in-bid-to-boost-sales-2016-5.jpg" alt="A man walks past the logo of Swiss drugmaker Novartis AG in front of a plant in Basel October 25, 2011. REUTERS/Arnd Wiegmann" data-mce-source="Thomson Reuters" data-mce-caption="A man walks past the logo of Swiss drugmaker Novartis AG in front of a plant in Basel" />According to a copy of one of the documents posted on the NGO's website, the embassy's second in command, Andr&eacute;s Fl&oacute;rez, suggests that "the direct link that exists between a significant group of members of Congress and the pharmaceutical industry in the United States, the case of GLIVEC is susceptible to escalate." He goes on to suggest it could impact funding of the ongoing peace process, as well as free trade treaties.</p>
<p>In another cable, Fl&oacute;rez, says that Everett Eissenstat, Chief of the International Trade Counsel for the US Senate Committee on Finance, "could jeopardize" peace funding.</p>
<p>"Mr Elissenstat mentioned that although Novartis is not an American company, the US pharmaceutical industry is very worried by the fact that such a case might become a precedent that could be applied for any patent in any industry," the letter said.</p>
<p>On Monday, 122 health, trade, and intellectual property international experts defended the Colombian government's right to issue a compulsory license on imatinib in a letter to Colombian President Juan Manuel Santos.</p>
<p>"We affirm that international law and policy support Colombia's right to issue compulsory licenses on patents in order to promote public interests including access to affordable medicines," the experts wrote. "When a pharmaceutical company uses a patent to exclude competition, it can charge much higher prices."</p>
<p>In a letter sent by the Colombian branch of Novartis, the company said the conditions in the market have actually reduced the price of imatinib making the government's decision unjustified. They insisted that they have the right to exclusively sell the medicine in Colombia until 2018.</p>
<p>"The imposition or the threat to impose a public interest declaration that would lead to a compulsory license shouldn't be used as a mechanism to enforce a price negotiation," the company said. "None of the factors that would justify this are present."</p>
<p>Meanwhile, health minister Gaviria said Novartis had the chance to negotiate and now the government has the right to enforce its decision.</p>
<p>"It is a complex step, we have checked all the international treaties, we have the autonomy to use these administrative tools," he said. "We have the authority to do it, we are willing to do so."</p><p><a href="http://www.businessinsider.com/colombias-battle-with-novartis-2016-5#comments">Join the conversation about this story &#187;</a></p> <p>NOW WATCH: <a href="http://www.businessinsider.com/bud-light-dilly-dilly-viral-commercial-super-bowl-campaign-2017-12">What 'Dilly Dilly' means — and how Bud Light came up with its viral campaign</a></p> http://www.businessinsider.com/abbvie-venetoclax-approval-2016-4The FDA just approved a new cancer drug — here's what you need to know about it (GILD)http://www.businessinsider.com/abbvie-venetoclax-approval-2016-4
Mon, 11 Apr 2016 13:47:00 -0400Lydia Ramsey
<p><img style="float:right;" src="http://static6.businessinsider.com/image/566202f28430761a008b7921-424-318/rbcs.jpg" alt="red blood cells" data-mce-source="Flickr/terrance stearns" data-mce-caption="Red blood cells" data-link="https://www.flickr.com/photos/figgy/2956281142/in/photolist-5veHow-id9MLt-id9Qxy-qBNtPV-4hYV3G-id9s8J-a86Si4-4AiMQM-4AiKLt-id9BUC-id9Ljw-id9WDP-id9A6M-id9MwG-4zGHgp-id9Hb8-p66r9U-4i47NB-ac8Qsa-rswunF-aRbxPX-7hNNNB-9RM2Az-bmtmdf-kh6V65-9RM36c-kh5dAQ-dr9B1t-4p2KR3-kueFS2-2GmX1-9JGkXz-sR5bT-5fAeTJ-4B3tfA-2abxc-sXxXU-7CuEzj-f4YmHQ-7JLcvm-sR59W-5ZcEji-7dpZGw-5ZcB9R-5ZgLqb-5ZgR61-9T29LR-5ZgQN1-9T29VX-9Vy4oa" /></p><p>The FDA just approved a brand new kind of cancer drug.&nbsp;</p>
<p>The drug, called venetoclax, <a href="http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm495253.htm">was approved</a> to treat a subset&nbsp;of people with one of the most common forms of adult&nbsp;leukemia, a type of cancer that affects the blood and bone marrow. It's&nbsp;made by drugmaker AbbVie.</p>
<p>Specifically, the drug&nbsp;targets those with chronic lymphocytic leukemia (CLL)&nbsp;who also&nbsp;have a precise genetic tweak (called a 17p deletion) and&nbsp;have <span>already been through</span>&nbsp;at least one other course of treatment.&nbsp;</p>
<h2>Here's what you need to know about the drug's approval:</h2>
<ul>
<li>Venetoclax, which will&nbsp;also go by the name Venclexta, is taken orally.</li>
<li>In a&nbsp;phase 2 study, which looked at 106 people with CLL and the specific genetic tweak, about 80% of patients experienced partial or complete remission by the end of the five-week period. Doses were increased over the five weeks from 20 mg to 400 mg.</li>
<li>Venetoclax is&nbsp;the first FDA-approved treatment to target something&nbsp;called "BCL2," a protein that helps cancer cells grow. Especially in patients with CLL, the BCL2 gene is overexpressed, meaning it makes&nbsp;too many copies of the protein. Venetoclax goes in and inhibits that protein,&nbsp;which gives healthy cells room to grow and keeps cancer cells from growing out of control.</li>
<li>That's especially helpful for those with the 17p deletion genetic tweak, because the mutation <a href="http://www.patientpower.info/what-exactly-does-a-17p-deletion-mean">makes it harder</a> for people with CLL to suppress cancer growth.</li>
<li>Michael Severino, AbbVie's Chief Scientific Officer told Business Insider that by inhibiting the BCL2 protein, they're able to avoid killing other cells (think: chemotherapy, which tends to target a wider range of cells).&nbsp;</li>
<li>Severino said the goal is to get venetoclax approved beyond just people who have CLL and&nbsp;the specific genetic tweak. Within CLL, there are some forms of the cancer that progress quicker than others. The genetic mutation, in particular, is associated&nbsp;with&nbsp;the more aggressive versions.</li>
</ul><p><strong>RELATED:&nbsp;<a href="http://www.businessinsider.com/abbvie-vice-president-of-oncology-development-gary-gordon-2015-12" >Here’s what the future of cancer treatment looks like</a></strong></p>
<p><strong>SEE ALSO:&nbsp;<a href="http://www.businessinsider.com/studies-of-vaginal-ring-to-prevent-hiv-2016-2" >Two new studies suggest this tiny device could help prevent HIV — with one major caveat</a></strong></p>
<p><a href="http://www.businessinsider.com/abbvie-venetoclax-approval-2016-4#comments">Join the conversation about this story &#187;</a></p> <p>NOW WATCH: <a href="http://www.businessinsider.com/better-memory-4-minute-guide-kevin-horsley-2018-2">A memory champion who has memorized 10,000 digits of pi reveals how to improve your memory in minutes</a></p> http://www.businessinsider.com/pharmaceutical-company-stock-up-over-300-2016-3This pharmaceutical company got great news about its cancer-fighting drug, and the stock skyrocketed over 400% (CPXX)http://www.businessinsider.com/pharmaceutical-company-stock-up-over-300-2016-3
Tue, 15 Mar 2016 10:33:20 -0400Bob Bryan
<p><span>A small biopharmaceutical company just got great news on a new drug, and the stock is up by a staggering amount.</span></p>
<p><span>Celator Pharmaceuticals <a href="http://ir.celatorpharma.com/releasedetail.cfm?ReleaseID=960543">announced late Monday</a> that its drug <span>Vyxeos, which</span> treats a type of blood cancer, tested well and was being prepared for submission to the Food and Drug Administration.</span></p>
<p><span>"<span>Based on these results the company expects to submit a New Drug Application (NDA) for VYXEOS with the </span>US Food and Drug Administration<span> (FDA) later this year and submit a Marketing Authorization Application (MAA) with the </span>European Medicines Agency<span> (EMA) in the first quarter of 2017," a release from the company said.</span></span></p>
<p><span><span>Vyxeos shows positive results in reducing mortality rates over 60 days in patients with h</span></span>igh-risk acute myeloid leukemia, or AML, to 13.7% from 21.2% in the control.</p>
<p>The stock shot up in response to the news, opening up by over 386% on Tuesday. In the last half-hour of trading it then found another leg up, trading up as much as 437% at $9.03.</p>
<p>While Celator is a small company, its market cap is now just under $300 million, and an increase above 400% for any stock in a single day is staggering, no matter the size.</p>
<p>This is huge news for investors and might be even better news for patients with AML.</p>
<p><img src="http://static5.businessinsider.com/image/56e8665f910584e73c8b6701-1098-710/screen shot 2016-03-15 at 3.45.18 pm.png" alt="Screen Shot 2016 03 15 at 3.45.18 PM" data-mce-source="Google Finance" /></p>
<p><em><strong><a href="https://twitter.com/bespokeinvest/status/709738424362786816">(h/t Bespoke Investment Group)</a></strong></em></p><p><strong>SEE ALSO:&nbsp;<a href="http://www.businessinsider.com/r-valeant-cuts-2016-revenue-forecast-2016-3" >Valeant is down over 30%</a></strong></p>
<p><a href="http://www.businessinsider.com/pharmaceutical-company-stock-up-over-300-2016-3#comments">Join the conversation about this story &#187;</a></p> <p>NOW WATCH: <a href="http://www.businessinsider.com/microsoft-president-brad-smith-davos-interview-trump-us-cant-get-too-isolationist-2018-1">Microsoft President Brad Smith says the US shouldn't get 'too isolationist'</a></p> http://www.businessinsider.com/r-new-kind-of-designer-immune-cells-clear-babys-leukaemia-2015-11Doctors used a gene-editing tool to cure a baby with untreatable leukemiahttp://www.businessinsider.com/r-new-kind-of-designer-immune-cells-clear-babys-leukaemia-2015-11
Thu, 05 Nov 2015 15:51:00 -0500Kate Kelland
<p><img style="float:right;" src="http://static4.businessinsider.com/image/563ba6635afbd3f62d8b4569-450-300/new-kind-of-designer-immune-cells-clear-babys-leukaemia.jpg" alt="Baby Layla (L) is seen with her parents, Lisa and Ashleigh, and her older sister Reya at Great Ormond Street Hospital (GOSH) in London in this November 4, 2015 handout photo by the hospital released on November 5, 2015. REUTERS/Great Ormond Street Hospital/Handout via Reuters." border="0" /></p><p></p>
<p>LONDON (Reuters) &mdash; A baby whom doctors thought almost certain to die has been cleared of a previously incurable leukemia in the first human use of an "off-the-shelf" cell therapy from Cellectis that creates designer immune cells.</p>
<p>One-year-old Layla had run out of all other treatment options when doctors at Britain's Great Ormond Street Hospital (GOSH) gave her the highly experimental, genetically edited cells in a tiny 1-milliliter intravenous infusion.</p>
<p>Two months later, she was cancer-free and she is now home from the hospital, the doctors said at a briefing about her case in London on Wednesday.</p>
<p>"Her leukemia was so aggressive that such a response is almost a miracle," said Paul Veys, a professor and director of bone marrow transplant at GOSH who led the team treating Layla.</p>
<p>"As this was the first time that the treatment had been used, we didn't know if or when it would work, so we were over the moon when it did."</p>
<p>The gene-edited cell treatment was prepared by scientists at GOSH and University College London (UCL) together with the French biotech firm Cellectis, which is now funding full clinical trials of the therapy due to start next year.</p>
<p>It is designed to work by adding new genes to healthy donated immune cells known as T-cells, which arm them against leukemia.</p>
<p>Using a gene-editing technology called TALEN, which acts as "molecular scissors," specific genes are then cut to make the T-cells behave in two specific ways: First, they are rendered invisible to a powerful leukaemia drug that would usually kill them, and second they are reprogrammed to only target and fight against leukemia cells.</p>
<p>Other drugmakers, including Novartis, Juno Therapeutics and Kite Pharma, have tested genetically modified T-cells extracted from an individual patient. However, this is the first time cells from a healthy donor have been used in a process that could lead to a ready off-the-shelf supply for use in multiple patients.</p>
<p>Some scientists have questioned Cellectis' approach because of potential problems with patients rejecting foreign cells.</p>
<p>But the French biotech, working with the U.S. giant Pfizer as well as Novartis, believes its method is faster and cheaper than creating single patient-specific gene therapies.</p>
<p>Results from Layla's case were due to be presented at the American Society of Hematology's annual meeting in Orlando on Wednesday.</p>
<p>"This is a landmark in the use of new gene engineering technology and the effects for this child have been staggering," said Waseem Qasim, a professor of Cell and Gene Therapy at UCL and immunologist at GOSH who worked on her medical team.</p>
<p>If the success in this case is sustained and replicated in other patients, he said, the therapy "could represent a huge step forward in treating leukaemia and other cancers."</p>
<p>Matt Kaiser, head of research at the leukemia and lymphoma charity Bloodwise, said that while the concept of editing immune cells to recognize and hunt out leukemia cells is "very exciting," patients and their families should note that the technique is still in the very early stages of development.</p>
<p>"We need to establish whether it can offer a long-term cure, whether there are any side effects and which patients are most likely to benefit from it," he said.</p><p><strong>SEE ALSO:&nbsp;<a href="http://www.businessinsider.com/editas-wants-to-use-crispr-by-2017-2015-11" >This startup wants to start using controversial CRISPR gene-editing technology in people by 2017</a></strong></p>
<p><strong>DON'T MISS:&nbsp;<a href="http://www.businessinsider.com/what-is-price-gouging-2015-11" >Turing Pharmaceuticals claims no patients are actually paying the full $750 per pill for Daraprim — here's who foots the bill instead</a></strong></p>
<p><a href="http://www.businessinsider.com/r-new-kind-of-designer-immune-cells-clear-babys-leukaemia-2015-11#comments">Join the conversation about this story &#187;</a></p> <p>NOW WATCH: <a href="http://www.businessinsider.com/suki-kim-north-korea-sent-hundreds-cheerleaders-olympics-2018-2">Why North Korea sent hundreds of cheerleaders to the Olympics</a></p> http://www.businessinsider.com/scientists-create-a-cancer-drug-that-helps-the-body-fight-the-disease-naturally-2014-6Scientists Create A Cancer Drug That Helps The Body Fight The Disease Naturallyhttp://www.businessinsider.com/scientists-create-a-cancer-drug-that-helps-the-body-fight-the-disease-naturally-2014-6
Thu, 12 Jun 2014 07:24:00 -0400Sarah Knapton
<p><img src="http://static4.businessinsider.com/image/52c71f6d69bedd5f160e77c7-1000-649/shutterstock_141299491.jpg" border="0" alt="Cancer cells" /></p><p>A pill which boosts the body&rsquo;s natural defences could help fight off all cancers and stop them ever returning, scientists believe.</p>
<p>&lsquo;Delta-inhibitors&rsquo; were already known to help leukemia patients, but researchers were amazed to find they also work on a whole range of other cancers.</p>
<p>The drugs, which are taken orally as a pill, were so successful in leukaemia trials that the control group, who were taking placebos, were immediately switched to the medication on ethical grounds.</p>
<p>Now, scientists at UCL and Cambridge University have discovered that the same &lsquo;delta inhibitors&rsquo; are also effective against lung, pancreatic, skin and breast cancers, and probably many more.</p>
<p>Cancer suppresses the immune system by producing an enzyme called &lsquo;p100delta&rsquo; which tells it to power down, making it difficult for the body to fight the disease. The drugs &lsquo;inhibit&rsquo; that enzyme, allowing the immune system to attack tumor cells.</p>
<p>The added benefit is that once the body has learned to fight off the cancer, it has in-built immunity, so that the disease can never return, unlike if it had been killed by chemotherapy.</p>
<p>Although the study was conducted in mice, researchers are confident it would work in humans and are hopeful that human trials will begin soon.</p>
<p>&ldquo;This helps your own immune system fight off the cancer better. The good guys win. And it seems to work on all cancers,&rdquo; said study co-leader Professor Bart Vanhaesebroeck of the UCL Cancer Institute, who first discovered the p110 delta enzyme in 1997.</p>
<p>&ldquo;It will work to a certain extent on its own, if the tumour is not too big, but it would be very effective after surgery, to prevent spreading.</p>
<p>&ldquo;So it is very exciting. We have shown that blocking &lsquo;p110delta&rsquo; also has the remarkable effect of boosting the body&rsquo;s immune response against leukemia's as well as other cancers.&rdquo;</p>
<p>The team showed that inhibiting the enzyme in mice significantly increased cancer survival rates across a broad range of tumour types, both solid and blood cancers.</p>
<p>Mice given the drug survived breast cancer for almost twice as long.</p>
<p>Their cancers also spread significantly less, with far fewer and smaller tumors developing. Survival after surgical removal of primary breast cancer tumours was also vastly improved, which has important clinical implications for stopping breast cancer from returning following surgery.</p>
<p>And they discovered that the immune system &lsquo;remembers&rsquo; the cancer and can fight it off completely again. Mice who were given cancer a second time all survived.</p>
<p>&ldquo;Our work shows that delta inhibitors can shift the balance from the cancer becoming immune to our body&rsquo;s defences towards the body becoming immune to the cancer,&rdquo; said study co-leader Dr Klaus Okkenhaug of the Babraham Institute at Cambridge University.</p>
<p>&ldquo;This provides a rationale for using these drugs against both solid and blood cancers, possibly alongside cancer vaccines, cell therapies and other treatments that further promote tumour-specific immune responses.&rdquo;</p>
<p>The drugs are already being used in clinical trials and have been granted Breakthrough Therapy status by the Federal Drugs Agency in the US, which means their development has been speeded up.</p>
<p>They could be available within just a few years if approved by European regulators and the National Institute of Clinical Excellence.</p>
<p>Professor Nic Jones, Cancer Research UK&rsquo;s chief scientist and director of the Manchester Cancer Research Centre, said: &ldquo;Treatments that train the immune system to recognize and kill cancer cells are showing huge promise in several types of cancer.</p>
<p>&ldquo;This new finding, although only at an early stage, offers the potential to develop more treatments that can do this in many more cancers, including ones that have real need for more effective treatments such as pancreatic cancer.&rdquo;</p>
<p>The study was published in the journal Nature and funded by Cancer Research UK, the Biotechnology and Biological Sciences Research Council and the Wellcome Trust</p>
<p><img class="nc_pixel" src="https://pixel.newscred.com/px.gif?key=YXJ0aWNsZT0xNDA1YmMwZjBmMzAzNTFkZWVlYmI5Zjk3NGE3OGViZiZub25jZT04N2ZlNmVmZC03ODViLTRiZTYtOTk3MS0yYjcwZGUyNjY0OWYmcHVibGlzaGVyPTczMGViODZhYjU5ZjBkNDE5MjZhYzY1YjAxZjgzZTJm" border="0" alt="" width="1" height="1" /></p><p><a href="http://www.businessinsider.com/scientists-create-a-cancer-drug-that-helps-the-body-fight-the-disease-naturally-2014-6#comments">Join the conversation about this story &#187;</a></p> http://www.businessinsider.com/university-of-memphis-qb-played-piano-at-st-judes-2013-7University Of Memphis QB Played Piano For Children at St. Jude's Hospitalhttp://www.businessinsider.com/university-of-memphis-qb-played-piano-at-st-judes-2013-7
Fri, 26 Jul 2013 16:00:00 -0400Philip Johnson
<p>University of Memphis quarterback Jacob Karam was playing piano for the kids at St. Jude's Children's Research Hospital in Memphis on Monday when&nbsp;11-year-old Breanna Bercegeay sat down for a heartwarming duet.&nbsp;</p>
<p>A YouTube video captures the pair singing Jessie J's "Price Tag." According to "<a href="https://www.facebook.com/CheeringForBreanna">Cheering for Breanna</a>," a Facebook page dedicated Bercegeay, she was diagnosed with a<span>cute myeloid leukemia (AML) on June 4, 2013. According to the YouTube description, the pair 10 songs in all.</span></p>
<p>Karam is a senior at Memphis and will be the Tigers starting quarterback this year. He threw 14 touchdowns last season, but his piano skills might be even more impressive. His key strokes and Bercegeay's pretty voice make for an impressive duo (video via <a href="http://www.huffingtonpost.com/2013/07/25/jacob-karam-duet_n_3653765.html">Huffington Post</a>):</p>
<p><iframe width="620" height="335" frameborder="0" src="//www.youtube.com/embed/sNLsAbBWl_s"></iframe></p><p><a href="http://www.businessinsider.com/university-of-memphis-qb-played-piano-at-st-judes-2013-7#comments">Join the conversation about this story &#187;</a></p> http://www.businessinsider.com/liz-smith-may-have-just-published-a-premature-obituary-for-nora-ephron-2012-6Author And Screenwriter Nora Ephron Dead At 71http://www.businessinsider.com/liz-smith-may-have-just-published-a-premature-obituary-for-nora-ephron-2012-6
Tue, 26 Jun 2012 20:26:00 -0400Aly Weisman
<p><img style="float:right;" src="http://static5.businessinsider.com/image/4d24edeecadcbbc441100000-364-272/nora-ephron.jpg" border="0" alt="Nora Ephron" width="364" height="272" /></p><p></p>
<p><strong><em>UPDATED</em></strong></p>
<p>Family members have confirmed earlier reports that <strong>Nora Ephron</strong>&nbsp;died today. She was 71.</p>
<p>Ephron is famous for writing screenplays such as "Silkwood," "When Harry Met Sally," "Sleepless in Seattle," and more recently "Julie and Julia."</p>
<p>Her son,&nbsp;Jacob Bernstein, told the <a href="http://www.businessinsider.com/blackboard/new-york-times" class="hidden_link">New York Times</a> <a href="http://www.nytimes.com/2012/06/27/movies/nora-ephron-essayist-screenwriter-and-director-dies-at-71.html" target="_blank">she died from pneumonia</a> which was triggered by leukemia.</p>
<p>Ephron was married three times. Her second marriage was to journalist&nbsp;<strong>Carl Bernstein</strong>&nbsp;of Watergate fame, with whom she has two sons.</p>
<p>The couple's divorce inspired Ephron's popular 1983 novel "Heartburn," which was later turned into a film of the same name starring&nbsp;<strong>Jack Nicholson</strong>&nbsp;and&nbsp;<strong>Meryl Streep</strong>.</p>
<p>Ephron has been married to her current husband, screenwriter&nbsp;<strong>Nicholas Pileggi</strong>, since 1987.</p>
<p>Developing...</p>
<p>Earlier, columnist <strong>Liz Smith</strong>&nbsp;published a remembrance of her friend, writer and filmmaker<strong>&nbsp;Ephron</strong>, who has reportedly passed away.</p>
<p>The piece was posted on Smith's <a href="http://www.wowowow.com/liz-smith/liz-smith-on-nora-ephron/" target="_blank">WowOWow The Women on the Web</a> site and ended with, "I won&rsquo;t say, 'Rest in peace, Nora' &ndash; I will just ask 'What the hell will we do without you?' Maybe. I hope. Nora will answer.&nbsp; Nora, we ARE&nbsp; all basket cases.&nbsp; There was no one like you.'"</p>
<p>But Ephron's reps told the <em>New York Observer</em> "<a href="http://observer.com/2012/06/liz-smith-seems-to-eulogize-nora-ephron-directors-camp-wont-confirm-rumors/" target="_blank">no comment</a>" on her current health status, while her publisher Knopf tells the <em><a href="http://www.businessinsider.com/blackboard/new-york-times" class="hidden_link">New York Times</a></em> that Ephron is still alive.</p>
<p>And moments ago, <a href="http://www.businessinsider.com/blackboard/the-daily" class="hidden_link">The Daily</a> Beast/Newsweek West Coast Editor<strong> Kate Aurthur</strong> tweeted:</p>
<p><img src="http://static5.businessinsider.com/image/4fea29f0eab8ea8f0a000011/kate-aurthur-nora-ephron-tweet.png" border="0" alt="Kate Aurthur Nora Ephron Tweet" /></p>
<p><img src="http://static4.businessinsider.com/image/4fea2bca69beddfc28000002/kate-aurthur-nora-ephron-tweet.png" border="0" alt="Kate Aurthur Nora Ephron Tweet" /></p><p><a href="http://www.businessinsider.com/liz-smith-may-have-just-published-a-premature-obituary-for-nora-ephron-2012-6#comments">Join the conversation about this story &#187;</a></p>